Thoracic (Pulmonary) Sarcoidosis Imaging: Practice Essentials, Radiography, Computed Tomography

The Lung Institute

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The Lung Institute provides comprehensive diagnostics and treatments to patients with illnesses that affect the lungs and breathing, including lung cancer, chronic obstructive pulmonary disease, cystic fibrosis, asthma, interstitial lung disease, bronchiectasis, benign and malignant neoplasms, and lung transplantation.

The Lung Institute has developed a comprehensive program across specialties with one site for virtually all aspects of ambulatory pulmonary patient care, including a full pulmonary function testing lab, complete radiology services, echocardiography, laboratory, and nuclear medicine services.

Our physicians and surgeons are national and international experts in lung disease. Together, they offer comprehensive services, including highly specialized care to patients.

Pipeline Report 2024: See The Life-changing Therapies Showing Promise

As another U.S. Election approaches and uncertainty looms over the bio-pharma world, the horizon remains bright with late-stage therapies showing promising results and transformative potential. Amid the evolving landscape, novel mechanisms, advanced formulations and strategic R&D approaches are setting the stage for significant treatment paradigm shifts.

In endocrinology, several next-generation therapies are advancing rapidly, signaling a shift toward greater convenience. Eli Lilly's orforglipron, an oral GLP-1 agonist, shows promise in obesity and diabetes with the convenience of daily dosing. Amgen's MariTide, targeting both GIPR and GLP-1R, could become a significant player in the weight-loss market, though safety concerns remain. Novo Nordisk is also pushing forward with oral semaglutide, expanding its reach into both obesity and Alzheimer's disease.

In dermatology, breakthrough therapies are making significant strides. Johnson & Johnson's JNJ-2113 offers an innovative oral option for psoriasis, with blockbuster potential. Sanofi's amlitelimab is poised to redefine atopic dermatitis treatment, especially as Dupixent nears the end of its exclusivity. Moonlake's sonelokimab is showing promise in addressing the unmet needs of hidradenitis suppurativa.

Cardiovascular drug development once again becomes an industry focus with several key therapies advancing toward commercialization to address significant unmet needs. Ionis's olezarsen shows strong potential in lowering triglycerides for rare disease familial chylomicronemia syndrome, with more eyes on a potential expansion into the lucrative hypertriglyceridemia market. BridgeBio's acoramidis, a novel TTR stabilizer, has shown impressive reductions in cardiovascular events and could challenge Pfizer's Vyndaqel market dominance in ATTR-CM. Meanwhile, NewAmsterdam Pharma's obicetrapib is generating excitement with its revival of the CETP inhibitor class to significantly impact lipid management.

As usual, oncology continues to be an active R&D space, but this time we are highlighting specialty oncology companies tackling niche patient populations with first-in-class drugs. Jazz Pharmaceuticals' zanidatamab is poised to be a first-in-class HER2-targeted bispecific antibody for biliary tract cancer, showing strong clinical data, though it will face competition from Daiichi Sankyo's Enhertu. Syndax's revumenib targets a difficult-to-treat leukemia subgroup and holds strong potential for expansion into other patient populations. Meanwhile, Merus' zenocutuzumab, an NRG1 fusion-targeted therapy for lung and pancreatic cancer, is garnering industry attention due to promising data and an FDA priority review.

In rare diseases, Johnson & Johnson's nipocalimab for myasthenia gravis shows strong efficacy for a potential approval and a broader application in autoimmune diseases, though faces stiff competition from other FcRn inhibitors. Novo Nordisk's Mim8 for hemophilia A could challenge Roche's blockbuster Hemlibra by offering more potent dosing and patient-friendly delivery. Solid Biosciences' SGT-003 gene therapy in Phase II for Duchenne muscular dystrophy is showing promise in early trials, particularly for patients unresponsive to existing treatments.

In pulmonology, all eyes are on Merck's sotatercept and its early 2025 earnings results in pulmonary arterial hypertension — set to speak to ultra-high expectations for the potentially "game-changing" drug. In gastroenterology, J&J's IL-23 Tremfya awaits an approval decision in Crohn's disease to unlock its blockbuster potential in inflammatory bowel disease. And, finally, in the world of epilepsy, French firm Crossject is gearing up for an emergency use authorization for Zepizure, a needle-free autoinjector for emergency use during an epilepsy crisis. The EUA is expected to set the product up nicely for subsequent commercial approvals.

Agents profiled in this report were chosen in consultation with experts from InThought Research and Ipsos as well as research platforms Adis Insight (with Credit Suisse Financial data) and Mindgram. Analyses of featured products include clinical data, revenue forecasts, expected launch dates and likelihood of success, updated as of September 18, 2024.

Credit: Getty Images / Peterschreiber.Media ENDOCRINOLOGY

Drug: Orforglipron

Company: Eli Lilly

Indication: Obesity

Stage: Phase III

Sales estimate: $3 billion by 2030 (diabetes and obesity) — Credit Suisse

Background: Orforglipron, Lilly's next-gen, once-daily oral GLP-1 agonist, is expected to generate pivotal data in 2025 across multiple Phase III trials, including the ACHIEVE-1, -2 and -3 glycemic control studies and ATTAIN-1 and -2 for weight loss and cardiovascular risk. Early Phase II results showed promising weight reduction between 8.6% and 14.7% at 26 and 36 weeks, alongside significant HbA1c reductions in T2D patients.

InThought: Unlike Novo's Rybelsus (semaglutide), which requires fasting and complex formulation, orforglipron's ease of use without food restrictions and formulation makes it far more accessible, especially for mass production. The strong Phase II data on weight loss and cardiometabolic improvements, combined with the drug's favorable safety profile, position it as a game-changer, pending Phase III confirmation. — Leon Henderson-MacLennan, medical adviser

Ipsos: Following orforglipron positive Phase II results, Phase III data are eagerly anticipated as data also suggest that orforglipron has a good tolerability profile versus other oral obesity agents in development — and, as a daily oral medication, it may offer a more convenient treatment option compared to injectable therapies. — Ramya Logendra, director, Global Obesity & Cardiometabolic Disease Monitors

Drug: MariTide(formerly AMG 133)

Company: Amgen

Indication: Obesity

Stage: Phase II

Sales estimate: $2.2 billion by 2030 — Credit Suisse

Background: MariTide is Amgen's subQ antibody peptide conjugate that acts as both a GIPR antagonist and GLP-1R agonist, reducing appetite and decreasing fat accumulation. Early Phase I results showed mean body weight reductions, ranging from -7.2% at the lowest dose (120mg) to -14.5% at the highest dose (420mg) by day 85, with durable effects lasting up to 150 days. The highest dose was linked to high rates of nausea and study dropouts. Topline Phase II data is expected in late 2024, as Amgen actively plans a Phase III.

InThought: MariTide's early Phase I success and Amgen's strategic confidence in scaling its manufacturing capacity have sparked investor excitement, even though pivotal trials are still pending. The drug's potential to impact multiple comorbidities, including heart, kidney and liver disease, presents a compelling case for its broad therapeutic application. However, concerns about adverse events remain, given the Phase I's AE rate in higher doses. — Henderson-MacLennan

Ipsos: MariTide's early Phase I efficacy [mean weight loss of 14.5% at 420mg by day 85] is promising, drawing interest around its novel mechanism. This approach, plus its potential for once-monthly dosing and data suggesting a longer duration of action, makes MariTide another potential game-changer. More data from the ongoing Phase II trial is needed to confirm its safety profile and advantage over competitors. — Logendra

Drug: Oral semaglutide

Company: Novo Nordisk

Indication: Obesity & Alzheimer's disease

Stage: Phase III

Sales estimates: $8.2 billion (obesity); $5.2 billion (Alzheimer's) by 2030 — Credit Suisse

Background: Novo Nordisk's oral semaglutide, building on its label (as Rybelsus 3mg, 7mg, 14mg) in type 2 diabetes, is being studied at a higher 50mg pill for obesity with additional Phase III results (in late 2024/2025) expected to speak to a potential filing. In the OASIS 1 Phase IIIa study, a daily 50mg tablet showed a 15.1% weight reduction over 68 weeks. Curiously, separate findings suggest oral semaglutide has the potential to reduce dementia risk, drawing attention to Phase III trial results in Alzheimer's disease in 2025.

InThought: Oral semaglutide's expanded dosing options (from existing Rybelsus doses) provide a clear path to growth in obesity treatment, positioning it for continued success despite early concerns about Rybelsus uptake challenges. Interestingly, an electronic health record review out of University of Oxford — designed to see whether subcutaneous (SC) semaglutide could cause neurological problems — suggested that SC semaglutide was associated with a 48% decline in dementia when compared to Merck's Januvia (sitagliptin). EVOKE — an ambitious, Phase III trial — will provide us with the first robust data set for any semaglutide formulation in Alzheimer's disease.  — Henderson-MacLennan

Ipsos: With its established safety profile (through its use in Rybelsus for type 2 diabetes) and convenient oral administration, oral semaglutide has the potential to be a promising new therapy in the obesity market. Its successful launch hinges upon further positive trial results from the Phase III clinical development program and Novo Nordisk's ability to scale up its manufacturing capacity to meet demand for both the injectable and tablet forms. — Logendra

Credit: Getty Images / NEMES LASZLO/SCIENCE PHOTO LIBRARY DERMATOLOGY

Drug: JNJ-2113 / JNJ 77242113

Company: Johnson & Johnson

Indication: Psoriasis

Stage: Phase III

Sales estimate: $292 million by 2029 — Global Data

Background: JNJ-2113 is a novel oral IL-23R antagonist peptide that binds with high affinity to the IL-23R and has properties that allow it to be absorbed with oral dosing. Topline Phase IIb data from the FRONTIER-1 study showed positive PASI 75, 90 and 100 compared to placebo. Phase II data indicates that efficacy can be sustained for up to a year, with 75% of patients achieving PASI75 at week 16 with twice-daily dosing. Phase III ICONIC program — comparing JNJ-2113 to BMS' oral TYK2 inhibitor deucravacitinib — is expected to report results in 2025.

InThought: JNJ-2113 aims to expand its Tremfya franchise by offering an oral treatment for psoriasis targeting IL-23. Otezla set the stage for oral psoriasis treatments, but a massive unmet need for oral options remains. The ICONIC program is progressing ahead of schedule, and there is significant investor and dermatologist interest in JNJ-2113's potential for "biologic-like efficacy" that has revolutionized the psoriasis space. The oral peptide has potential to also be evaluated in other immune-mediated diseases. — Amanda Weyerbacher, senior principal

Ipsos: Hopes are high for J&J's novel IL-23, potentially the first oral in the therapy class. Topline Phase IIb efficacy (PASI 75, 90 and 100) data vs placebo lend credence to the MOA's efficacy in PsO. With multiple oral advanced therapies in development, how healthcare professionals employ novel treatments in a market well-served by injectables will be prominent areas of focus moving forward. — Erik Olson, SVP, healthcare NA

Drug: Amlitelimab

Company: Sanofi

Indication: Atopic dermatitis

Stage: Phase III

Sales estimates: $375 million by 2030 (AD); $842 million by 2030 (all indications) — Credit Suisse

Background: Amlitelimab is a monoclonal antibody targeting OX40-Ligand with potential to be a first- or best-in-class treatment for AD and other immune-mediated diseases. Phase IIb STREAM-AD trial data showed a significant improvement, with an average EASI score of 61% at week 16, nearly double that of the placebo. Phase III data is expected in 2025.

InThought: Sanofi's built a blockbuster franchise with its pipeline in a product, Dupixent, for which AD is its lead indication. It's planning its next steps as Dupixent's exclusivity ends in 2031. Sanofi is running a broad Phase III program for amlitelimab that includes a notable endpoint of EASI 100 and evaluation in adolescent patients. Its efficacy across a range of doses may reduce treatment burden with less frequent dosing. — Weyerbacher

Ipsos: Amlitelimab's early-stage STREAM-AD clinical trial data shows durable efficacy with a 12-week dosing schedule and exhibits a favorable safety profile — notably with the absence of fever and chills as side effects. If these initial findings are confirmed in late-stage trials, the extended dosing interval could provide a significant advantage in atopic dermatitis treatment, hopefully enhancing patient compliance further. — MamTing Thoo, research manager, global immunology monitors

Drug: Sonelokimab

Company: Moonlake Immunotherapeutics

Indication: Hidradenitis suppurativa (HS)

Stage: Phase III

Sales estimate: $563 million by 2030 — Credit Suisse

Background: Sonelokimab is an anti-IL-17 A/F Nanobody. Phase II data in HS, characterized by swollen bumps on the skin, showed 43% Hidradenitis Suppurativa Clinical Response (HiSCR75) at 120 mg versus about 15% with placebo at week 12. The 120 mg dose was chosen for further trials, with improvements increasing to 57% at week 24. Phase III VELA results are anticipated in mid-2025.

InThought: MoonLake will have to show a clinical profile for sonelokimab that meets the bar set by its IL-17 competitors such as Cosentyx and Bimzelx, as well as original mainstay Humira. But given the high unmet need for the burden of HS there is still room for therapeutic options. It is notable that the Phase III HS trials are using HiSCR75 as the primary endpoint, which could set a new standard of care in HS. — Weyerbacher

Ipsos: MoonLake's sonelokimab may have a familiar dual IL-17A/ F inhibition approach, but this next-generation therapy utilizes a novel class of therapeutic proteins from Sanofi's Nanobody technology platform. This unique small molecule allows highly efficient binding, which may not be accessible by conventional, larger molecules. Following positive 24-week Phase II data and a consistent safety profile, Phase III results will be highly anticipated. — Penny Robinson, research manager

Credit: Getty Images / Artpartner-images CARDIOVASCULAR

Drug: Olezarsen

Company: Ionis

Indication: Familial chylomicronemia syndrome (FCS)/ severe hypertriglyceridemia

Stage: Pre-registration (PDUFA December 2024)/Phase III

Sales estimate: More than $1 billion — Ionis' projections

Background: Olezarsen is an RNA-targeted investigational Ligand-Conjugated Antisense (LICA) medicine for people at risk of disease due to elevated triglyceride levels. In the Phase III Balance study, Olezarsen showed a statistically significant reduction of fasting triglycerides in patients with rare disease familial chylomicronemia syndrome (FCS). An initial approval in FCS is expected on December 19, 2024, with Phase III data in much larger and lucrative indication severe hypertriglyceridemia (sHTG) expected in late 2025.

InThought: Olezarsen did not only show strong triglyceride reduction in patients with FCS, it also showed a significant reduction in acute pancreatitis, an important comorbidity in these patients. Although the FCS patient population is small (1-13/1,000,000 in the U.S.), the drug's real blockbuster opportunity lies in the treatment of patients with severe hypertriglyceridemia (>3M patients in the U.S.). With its clinical trial program in this indication being fully enrolled, full data can be expected in the later half of 2025, setting Ionis up for its first penetration of a large indication. — Jasmien Roosenboom, senior principal

Drug: Acoramidis

Company: BridgeBio/Eidos Therapeutics

Indication: ATTR-CM

Stage: Pre-registration (PDUFA November 29, 2024)

Sale estimate: $1.8 billion by 2030 — Leerink

Background: Acoramidis — a novel TTR stabilizer — is being developed to treat ATTR-CM where cardiac amyloid fibrils infiltrate the heart, leading to restrictive cardiomyopathy and heart failure. While Pfizer's Vyndaqel (tafamidis) has been the only approved therapy since 2019, BridgeBio's acoramidis along with Alnylam's vutrisiran are expected to join the market by 2025. In its Phase III ATTRibute-CM trial, Acoramidis demonstrated a 42% reduction in all cause mortality (ACM) or cardiovascular hospitalization (CVH) at month 30.

InThought: Both BridgeBio's acoramidis and Alnylam's vutrisiran appear poised to capture some of Pfizer's ATTR-CM market share while also expanding the market due to under-recognition and underdiagnosis. Current estimates suggest around 47,000 diagnosed U.S. Patients, while other industry estimates range from 100,000-300,000. While acoramidis has shown promising results with a reduction in mortality risk, caution is advised in interpreting these results, particularly due to the unconventional "win ratio" approach used in its primary endpoint analysis. — Henderson-MacLennan

Ipsos: BridgeBio's acoramidis shows promise for ATTR-CM, with Phase III results [42% reduction in ACM or CVH events at month 30], surpassing both tafamidis and vutrisiran. The drug also demonstrated greater risk reduction and improved NT-proBNP and serum TTR levels compared to the placebo group. Higher serum TTR levels correlate with higher survival rates, supporting acoramidis' potential in the growing ATTR-CM market. — Joel Sandler, associate partner, healthcare, NA

Drug: Obicetrapib

Company: NewAmsterdam Pharma

Indication: Lowering LDL cholesterol

Stage: Phase III

Sales estimate: $1.4 billion by 2032 — GlobalData

Background: Obicetrapib is a small molecule cholesterol ester transfer protein (CETP) inhibitor with many pivotal trials — due to report throughout 2025 — assessing its efficacy in LDL-lowering for heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease (ASCVD). Preliminary results from the Phase III BROOKLYN study show a significant 36.3% LDL reduction at 84 days and 41.5% at 365 days, with improvements in HDL, non-HDL, Lp(a), ApoB and acceptable safety and tolerability profiles. The BROOKLYN (HeFH) and BROADWAY (HeFH and non-HeFH) Phase III trials are due to readout in late 2025.

InThought: The promising results of obicetrapib and the revival of the CETP inhibitor class are encouraging. With strong LDL-lowering performance and a favorable safety profile, obicetrapib could significantly impact lipid management. We anticipate U.S. And EU regulatory filings for LDL lowering by early 2026 and for major adverse cardiovascular events (MACE) by late 2026 or early 2027. In the first half of 2025 we also expect data out of the TANDEM trial of a fixed-dose combination of obi and ezetimibe in subjects on maximally tolerated lipid lowering therapy with HeFH, ASCVD, or elevated risk for ASCVD. — Henderson-MacLennan

Credit: Getty Images / KATERYNA KON/SCIENCE PHOTO LIBRARY ONCOLOGY

Drug: Zanidatamab

Company: Jazz Pharmaceuticals

Indication: HER2-positive biliary tract cancer

Phase: Pre-registration (PDUFA November 29, 2024)

Sales estimates: $100 million by 2030 (China) — Credit Suisse; more than $2 billion opportunity — Jazz's projections

Background: Zanidatamab is an investigational first-in-class bispecific HER2-targeted antibody, with a lead indication of previously-treated HER2+ biliary tract cancer (BTC). Data from its HERIZON-BTC-01 trial has demonstrated a sustained objective response rate of 41% and a 69% disease control rate. Median duration of response increased to 14.9 months with median overall survival of 15.5 months.

InThought: Jazz Pharmaceuticals' most compelling growth driver and its approaching approval with strong physician enthusiasm especially in biliary tract cancer. Entering a market with zero competition, especially in HER2+ patients where there is no direct marketed competitor. — Weyerbacher

Ipsos: While zanidatamab would be the first HER2-targeted agent with a specific label for BTC, it will face competition from Daiichi Sankyo's Enhertu, which received a broad HER2+ solid tumor-agnostic label last year. Enhertu's first-mover advantage and overall brand equity combine to create a fierce competitor for zanidatamab. — Eric Blouin, SVP , oncology, healthcare, NA

Drug: Revumenib

Company: Syndax

Indication: Acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL)

Stage: Pre-registration (PDUFA December 26, 2024)

Sales estimate: $707 million by 2033 — Global Data

Background: Syndax aims to launch revumenib in KMT2Ar acute leukemia, a difficult-to-treat subgroup accounting for approximately 10% of all AML or ALL patients. The regulatory application is supported by positive data from the pivotal Phase II AUGMENT-101 trial that met its primary endpoint at the protocol-defined interim analysis with a complete response (CR) or a CR with partial hematological recovery (CRh) rate of 23%.

InThought: Despite a three-month delay in regulatory review, Syndax is still on track to secure a first-in-class approval for this brand-new patient subset. The company plans to quickly expand into earlier treatment settings, additional patient populations and explore combination therapies with an anticipated >$4 billion market potential. Patients and physicians are excited to bring much-needed targeted therapies to this difficult-to-treat cancer. — Ashley Ingles, senior analyst

Ipsos: Although the discovery and "druggability" of the FLT3 pathway generated targeted therapies for specific relapsed/refractory (R/R) AML patient types, over half still have limited clinical options. Therefore, data from the AUGMENT-101 study are exciting to see; 2 in 3 patients responded, and almost a quarter saw a complete remission. While research shows associated genes rapidly developing resistance to revumenib, combination trials are already in place to address this. — Amy Butcher, senior director, oncology, healthcare, U.K.

Drug: Zenocutuzumab

Company: Merus

Indication: Non-small cell lung cancer (NSCLC) and pancreatic cancer

Stage: Pre-registration

Background: Zenocutuzumab is the first and only targeted therapy for patients with lung and pancreatic cancer that is positive for NRG1 (neuregulin 1 fusion) mutation. Phase I/II eNRGy trial results were shared at EMSO 2023 and showed an overall response rate of 37% in non-small cell lung cancer and 42% in pancreatic cancer in the monotherapy setting. Of all patients evaluated in the basket trial, 61% experienced a treatment emergent adverse event, but only 6% of these patients had an event that was grade 3 or 4.

InThought: FDA breakthrough designations leading to priority review was a big win for the Dutch-based biotech, which the investor community is keeping a close eye on. We'll need to watch for its success (and possible challenges) in testing for patients who express NRG1. The pending approval will also add momentum for the potential expansion of zenocutuzumab into other tumor types irrespective of NRG1 status. — Weyerbacher

Ipsos: Zenocutuzumab emerges as the most promising investigational drug for NRG1 fusion-positive cancers, a group of malignancies currently lacking approved targeted therapies and showing limited response to off-label treatments. Encouraging Phase I/II eNRGy trial results combined with FDA priority review highlight zenocutuzumab's potential as a first-to-market treatment for these rare molecular subtypes of cancer. — Agata Atkins, director, global oncology monitors

Credit: Getty Images / KATERYNA KON/SCIENCE PHOTO LIBRARY RARE DISEASE

Drug: Nipocalimab

Company: Johnson & Johnson

Indication: Myasthenia gravis (gMG)

Stage: Pre-registration (H2 2025)

Sales estimates: $561 million by 2030 (MG); $4.2 billion by 2030 (all indications) — Credit Suisse

Background: Nipocalimab is an FcRn inhibitor for rare long-term neuromuscular junction disease myasthenia gravis (gMG). Phase III (Vivacity-MG3) data showed gMG patients receiving nipocalimab, plus standard of care, improved by 4.70 points on the MG activities of daily living profile (MG-ADL), achieving superiority over placebo. After an August 2024 BLA submission, an approval decision is expected in H2 2025.

InThought: While it will be the third FcRn inhibitor to enter myasthenia gravis, nipocalimab is pursuing several indications, including larger indications such as rheumatoid arthritis with J&J suggesting peak sales potential of $5 billion across its indications. However, nipocalimab's success could be tempered by the increasing competition in the FcRn space — such as Argenx's Vyvgart and Immunovant's FcRn franchise, which offer a convenient subQ administration versus nipocalimab's IV route and will likely pursue overlapping indications. — Antoineen White, senior analyst

Ipsos: Phase III data, achieved through a broad population of gMG patients sustaining a response over six months, are unprecedented for an FcRn blocker. Alongside data indicating a favorable safety profile, they are particularly encouraging for those living with anti- MuSk+ and anti-LRP4+ forms of gMG, for which there are currently limited or no treatment options. — Hannah Brown, director, global neurology monitors

Drug: Mim8

Company: Novo Nordisk

Indication: Hemophilia A

Stage: Pre-registration (expected end 2025)

Sales estimate: $849 million by 2030 — Credit Suisse

Background: Mim8 is a bispecific monoclonal antibody similar to Roche's Hemlibra that is being developed for hemophilia A. Phase IIIa Frontier II data in hemophilia A showed the drug significantly reduced treated bleeding episodes. Novo Nordisk expects to file Mim8 for approval in early 2025. If the drug receives priority review, it could be approved by the end of 2025.

InThought: Mim8 is more potent than Roche's Hemlibra, which allows it to be delivered in a smaller dose with similar or improved efficacy compared to Hemlibra. Novo Nordisk will use its diabetes device expertise to bring Mim8 to the market with a convenient prefilled pen that is likely to be preferred by patients compared to Hemlibra. With Hemlibra sales of $4.7 billion in 2023, Mim8 has the potential to be a blockbuster if Novo Nordisk can convert a portion of Hemlibra patients to Mim8. — Chris Martin, senior principal

Ipsos: Its convenient once-weekly and once-monthly dosing combined with strong efficacy data could change the treatment landscape and give HCPs another non-factor replacement therapy option besides emicizumab. Mim8 may drive strong adoption and improve patient quality of life, while Novo Nordisk's expertise in the field further supports Mim8's potential to make a significant impact. — Felix Hey, senior research executive, global rare disease monitors

Drug: SGT-003

Company: Solid Biosciences

Indication: Duchenne muscular dystrophy

Stage: Phase II

Background: SGT-003 is a next-generation gene therapy for DMD using a novel capsid to deliver a microdystrophin gene (including the nNOS binding domain), which may improve muscle function and resilience. In its ongoing Phase I/II trial, Solid said the drug was well tolerated, with no serious adverse events, consistent with the IND-enabling NHP toxicology study. Trial expansion and patient population broadening are planned, with initial safety and three-month functional data expected by Q1 2025.

InThought: Solid is positioning SGT-003 as a secondary gene therapy option, particularly for patients with seropositivity to existing therapies. If safety results continue to show positive, paired with functional data that is comparable to currently approved therapies, SGT-003 may be a top contender for approval in upcoming years. — Lauren Jonas, senior analyst

OTHER Credit: Getty Images / MARHARYTA MARKO PULMONOLOGY

Drug: Sotatercept

Company: Merck

Indication: Pulmonary arterial hypertension

Stage: Phase III/launch

Sales estimate: $4.4 billion by 2030 — Credit Suisse

Background: Sotatercept (Winrevair), is a recombinant activin receptor type IIA-Fc fusion protein targeting pulmonary arterial hypertension (PAH). Approved in 2024, impressive outcomes data and real-world projections suggest the drug could redefine PAH management, drawing sharp focus on its first-year earnings results in early 2025. Sotatercept targets pulmonary vessel remodeling, making it a potential treatment for other forms of pulmonary hypertension, including PH due to left heart disease and PH-ILD, with subsequent Phase III readouts expected to generate interest.

InThought: Sotatercept's strong clinical data has positioned it as a potential game-changer in PAH treatment, with experts suggesting its disease-modifying potential due to its magnitude of effect. Expectations for significant market impact are further supported by a favorable ICER report, highlighting benefits such as extended life expectancy, reduced hospitalizations and lung transplant avoidance. — Henderson-MacLennan

Ipsos: In 2021, Merck acquired Acceleron for $11 billion, attracted by its PAH treatment, sotatercept. This reverse-remodeling agent improved clinical outcomes in preclinical and Phase II trials, and the Phase III STELLAR study confirmed its significant benefits, including an 84% reduction in death or non-fatal clinical worsening events. Merck will conduct Phase III trials (HYPERION and ZENITH) to expand its use in multiple therapy lines. — Sandler

Credit: Getty Images / Magicmine GASTROENTEROLOGY

Drug: Tremfya (guselkumab)

Company: Johnson & Johnson

Indication: Crohn's disease (CD)

Stage: Pre-registration

Sales estimate: $1.24B in Crohn's Disease by 2028 — Credit Suisse

Background: Tremfya is J&J's IL-23 blockbuster, initially approved for plaque psoriasis, but also expected to make blockbuster strides in inflammatory bowel disease (IBD). It just nabbed an approval in ulcerative colitis so now hopes are high for Crohn's disease (CD). In CD, Tremfya showed superiority over Stelara in the Phase III GALAXI study for endoscopic endpoints and clinical remission and also met both co-primary endpoints in the Phase III GRAVITI study at week 12.

InThought: Tremfya's long-standing claim in the psoriatic disease space now has the welcome opportunity to expand into IBD, and the field is ready given the outstanding momentum of IL-23s in this space, with many experts believing they will be effective as a first-line treatment. Potential to be first-in-class to offer options of both subcutaneous and intravenous induction therapy in CD. — Weyerbacher

Ipsos: J&J seeks to continue its IBD biologic heritage with Tremfya and to blunt revenue losses from biosimilar competition to Remicade and Stelara. In CD, J&J's IL-23 demonstrated superiority versus its own Stelara in endoscopic improvement and clinical remission data in the Phase III GALAXI 2 & 3 studies; it remains to be seen how Tremfya's clinical profile will be viewed relative to other IL-23s. — Olson

Credit: Getty Images / Science Photo Library – SCIEPRO NEUROLOGY

Drug: Zepizure

Company: Crossject

Indication: Epilepsy

Stage: Pre-registration

Background: Small French biotech Crossject is gearing up for regulatory filing of Zepizure (zeneo midazolam) for emergency and commercial use in the U.S. As well as for commercial use in the EU, for the treatment of status epilepticus. Zepizure is a needle-free autoinjector, developed to terminate seizures rapidly, and development was partially funded by the U.S. BARDA program, in order to stockpile the antiseizure drug for emergency use.

InThought: The proceeds of the BARDA agreement and stockpiling will set Crossject up for pursuing a commercial indication in the U.S. And the EU. With limited competition (Neurelis' nasal spray Valtoco and Neuraxpharm's Buccolam), Crossject can make a splash in the market and try to define a broad rescue indication for the drug. Of note, main pipeline competitors UCB (Staccato Alprazolam) and Aquestive (Libervant) are also still a few years out for launch in the adult population, giving Crossject the opportunity to shape the rescue medicine market. — Roosenboom

From the October 01, 2024 Issue of MM+M - Medical Marketing and Media

Division Of Pulmonary, Critical Care And Sleep Medicine

Fiberoptic Bronchoscopy and Interventional Pulmonary Medicine

A modern bronchoscopy suite with fluoroscopy is available for ambulatory and inpatient bronchoscopic procedures.

Fellows have ample opportunities to develop skills in performing bronchoalveolar lavage, endobronchial and transbronchial lung biopsies in addition to endobronchial ultrasound (EBUS) and endoscopic ultrasound (EUS) directed procedures using both linear and radial instruments in diagnostic evaluation and staging in patients with suspected intrathoracic malignancy.

Training in procedures such as PleurX catheter placement, talc pleurodesis, tracheal dilation, rigid bronchoscopy, thoracoscopy, endobronchial stenting, balloon bronchoplasty, argon photocoagulation (APC) and cryotherapy are performed when appropriate under the direction of board certified interventional pulmonology faculty. Percutaneous tracheostomies are performed at the bedside on patients with chronic respiratory failure under bronchoscopic guidance at both SSM Health Saint Louis University Hospital and Mercy Hospital St. Louis.

In addition, fellows gain experience in a number of other invasive diagnostic and therapeutic techniques used in pulmonary medicine such as pleuroscopy, pleural biopsy and the placement and management of chest tubes.

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